Effects of Thalidomide on Metabolism and Lifespan of Red Blood Cell in Patients With β-Thalassemia Major: A Post Hoc Analysis of a Randomized Controlled Trial.

PURPOSE: Recent studies have shown the thalidomide's therapeutic potential in treatment of patients with β-thalassemia major. However, the effect of thalidomide on metabolism and lifespan of red blood cells (RBCs) is rarely reported.

METHODS

This study was a post hoc analysis of a randomized controlled trial (Chinese Clinical Trial Registry, ChiCTR1800015702). One hundred patients with β-thalassemia major were randomly assigned 1:1 to treatment with a placebo or thalidomide. The primary outcomes were the differences in RBC lifespan, reticulocyte count, and peripheral nucleated RBC count of patients after treatment of 12 weeks. Other indicators of hemolytic reaction were also analyzed.

FINDINGS: Compared with the placebo group after treatment of 12 weeks, the thalidomide group showed a longer RBC lifespan (16.29 ± 6.42 vs 12.90 ± 4.98 days; P = 0.004), smaller mean corpuscular volume (68.34 ± 7.79 vs 78.01 ± 6.33 fl; P < 0.001), smaller mean corpuscular hemoglobin (21.62 ± 2.85 vs 24.68 ± 2.69 pg; P < 0.001), and lower lactate dehydrogenase (190.00 [148.00 - 305.00] vs 251.00 [199.20 - 327.80]; P = 0.014). Meanwhile, thalidomide significantly increased the RBC lifespan at 24 weeks (21.24 ± 8.30 days; P < 0.001) and 48 weeks (23.21 ± 8.42 days; P < 0.001) when compared with baseline (12.8 ± 6.0 days).

IMPLICATIONS: Thalidomide increases the RBC lifespan and reduces hemolytic reactions in patients with β-thalassemia major. Chinese Clinical Trial Registry identifier: ChiCTR1800015702.